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Phase IIIb, Open-label, Multi-center Study to Evaluate Safety, Tolerability and Efficacy of OAV101 Administered Intrathecally to Participants With SMA Who Discontinued Treatment With Nusinersen or Risdiplam

NCT05386680 · Status: COMPLETED · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 27

Last updated 2026-01-13

Study results available
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Summary

This was a Phase IIIb open-label, single arm, multi-center study to evaluate the safety, tolerability and efficacy of OAV101B in participants with SMA aged 2 to \<18 years after the discontinuation of treatment with nusinersen or risdiplam. The study aimed to enroll approximately 28 participants across each of 2 age brackets (2 to \<6 years, and 6 to \<18 years).

Conditions

Interventions

GENETIC

OAV101

Intrathecal administration of OAV101 at a dose of 1.2 x 10\^14 vector genomes, one time dose

Sponsors & Collaborators

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
2 Years
Max Age
17 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2023-01-12
Primary Completion
2024-11-29
Completion
2024-11-29
FDA Drug
Yes

Countries

  • United States
  • Australia
  • Belgium
  • Canada
  • France
  • Italy
  • Japan
  • Netherlands
  • Spain

Study Locations

More Related Trials

Entities

Diseases
Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT05386680 on ClinicalTrials.gov